Autologous haematopoietic stem-cell transplantation in multiple sclerosis

Summary Intense immunosuppression followed by autologous haematopoietic stem-cell transplantation has been assessed over the past few years as a possible new therapeutic strategy in severe forms of multiple sclerosis. Pioneering studies began in 1995, and since then, more than 400 patients worldwide have been treated with this procedure. Small uncontrolled studies show that about 60–70% of treated cases do not progress in the follow-up period of at least 3 years. Transplant-related mortality, which was 5–6% in the first reported series, has reduced in the past 5 years to 1–2%. Relapses dramatically decrease and inflammatory MRI activity is almost completely suppressed. Autologous haematopoietic stem-cell transplantation is associated with qualitative immunological changes in the blood, suggesting that, beyond its immunosuppressive potential, it could also have some beneficial effect for the resetting of the immune system. Patients with severe, rapidly worsening multiple sclerosis who are unresponsive to approved therapies could be candidates for this treatment, but its clinical efficacy has still to be shown in large, prospective, controlled studies.